Multiple Doses of AT-1501-A201 in Adults With ALS
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04322149 |
Recruitment Status :
Recruiting
First Posted : March 26, 2020
Last Update Posted : January 22, 2021
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
This is a Phase 2a, multi-center, open label, multiple dose study of AT-1501, a humanized monoclonal antibody antagonist to CD40LG. Approximately 54 adults with ALS will be enrolled into the study in the United States at up to 12 ALS treatment sites.
Participants will be enrolled into one of four ascending doses.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Amyotrophic Lateral Sclerosis | Drug: AT-1501 | Phase 2 |
This is a Phase 2a, multi-center, open label, multiple dose study of AT-1501, a humanized monoclonal antibody antagonist to CD40LG. Approximately 54 adults with ALS will be enrolled into the study in the United States at up to 12 ALS treatment sites.
Four ascending doses of AT-1501 will be administered as an IV infusion to sequentially enrolling cohorts. Each participant will receive 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period.
The study is estimated to take 19 weeks for participants.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 54 participants |
Intervention Model: | Sequential Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 2a Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Multiple Doses of AT-1501 in Adults With ALS |
Actual Study Start Date : | October 16, 2020 |
Estimated Primary Completion Date : | September 2021 |
Estimated Study Completion Date : | October 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: AT-1501
4 sequential dose cohorts
|
Drug: AT-1501
AT-1501 monoclonal antibody targeting CD40LG given as an IV infusion |
- Safety and tolerability [ Time Frame: Approx. 5 Months ]Incidence of adverse events (AEs) as characterized by, changes in physical examination, vital signs, ECG, laboratory parameters, and C-SSRS from baseline

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- ALS diagnosed as possible, laboratory supported probable, probable, or definite as defined by revised El Escorial criteria.
- ALSFRS-R Aggregate score of 37 or greater
- No more than 24 months from diagnosis.
- Able and willing to give informed consent, follow trial procedures, and make multiple clinical site visits.
Exclusion Criteria:
- Any other central or peripheral nervous system disease that may interfere with the evaluation of ALS or its progression.
- Presence of a tracheostomy, or use of permanent assistive ventilation (ventilatory support for 23 hours per day or more).
- History of malignancy within the previous 5 years, except for localized non-melanoma skin cancers.
-
Abnormal function of the immune system resulting from:
- Clinical conditions affecting the immune system (e.g. HIV infection, agammaglobulinemia),
- Systemic administration of corticosteroids (PO/IV/IM) at a dose equivalent to 20 mg/day of prednisone for more than 14 consecutive days within 90 days prior to screening,
- Administration of anti-neoplastic and/or immunomodulating agents (e.g. TNF α antagonists or anti-B cell antibodies) or radiotherapy within 1 year prior to screening.
- Recipient of Stem Cell or Gene Therapy.
- Positive test for Hepatitis B surface antigen, Hepatitis C antibody, or HIV.
- History of deep venous thrombosis or pulmonary embolism.
- History of active substance abuse within the past 2 years;
- History of stroke, poorly controlled or significant cardiovascular disease, diabetes.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04322149
Contact: Jennifer Bartoshevich | 16033205141 | jbartoshevich@eledon.com |
United States, Arizona | |
Barrows Neurological Institute | Recruiting |
Phoenix, Arizona, United States, 85013 | |
Contact: Research Staff 602-406-4232 stephanie.Strong@dignityhealth.org | |
United States, California | |
University of California Irvine | Recruiting |
Orange, California, United States, 92868 | |
Contact: Research Staff 714-509-2661 eonv@hs.uci.edu | |
California Pacific Medical Center | Recruiting |
San Francisco, California, United States, 94109 | |
Contact: Research Staff 415-600-3758 EngelM@cpmcri.org | |
United States, Georgia | |
Augusta University | Recruiting |
Augusta, Georgia, United States, 30912 | |
Contact: Research Staff 706-721-2681 BQUARLES@augusta.edu | |
United States, Indiana | |
University of Indiana | Recruiting |
Indianapolis, Indiana, United States, 46202 | |
Contact: Research Staff 317-963-7382 sguingri@iu.edu | |
United States, Maryland | |
Johns Hopkins University Medical Center | Recruiting |
Baltimore, Maryland, United States, 21287 | |
Contact: Research Staff 410-955-8511 kriley15@jhmi.edu | |
United States, Massachusetts | |
Massachusetts General Hospital | Not yet recruiting |
Boston, Massachusetts, United States, 02114 | |
Contact: Research study Staff 617-643-7428 ZSCHEIER@mgh.harvard.edu | |
United States, New York | |
Hospital for Special Surgery (HSS) | Recruiting |
New York, New York, United States, 10021 | |
Contact: Research Staff 646-797-8592 HolzbergS@hss.edu | |
United States, Oregon | |
Providence Brain & Spine Institute | Not yet recruiting |
Portland, Oregon, United States, 97213 | |
Contact: Research Study Staff 503-962-1171 arlena.cummings@providence.org | |
United States, Texas | |
Houston Methodist Neurological Institute | Recruiting |
Houston, Texas, United States, 77030 | |
Contact: Research Staff 713-441-9120 rgapplegate@houstonmethodist.org |
Study Chair: | Steve Perrin, PhD | Anelixis Therapeutics, Inc. |
Responsible Party: | Anelixis Therapeutics, Inc. |
ClinicalTrials.gov Identifier: | NCT04322149 |
Other Study ID Numbers: |
AT-1501-A201 |
First Posted: | March 26, 2020 Key Record Dates |
Last Update Posted: | January 22, 2021 |
Last Verified: | January 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
ALS Lou Gehirg's Disease motor neuron disease motor neuron degeneration |
AT-1501 humanized blocking antibody to CD40LG CD40LG inhibitor monoclonal antibody |
Motor Neuron Disease Amyotrophic Lateral Sclerosis Neurodegenerative Diseases Nervous System Diseases Neuromuscular Diseases |
Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases |